To those who say "impossible, impractical, unrealistic," we say:

CHALLENGE ACCEPTED

We're relentless in our pursuit of new treatments because patients shouldn't have to wait for hope.

Rose, living with a type of acute hepatic porphyria

We are developing a new class of innovative medicines called

RNAi THERAPEUTICS

that we believe have the potential to transform the lives of people living with rare diseases.

Dane, living with hATTR amyloidosis

We are focused on diseases for which treatment options are limited or simply don't exist.

OUR ROBUST PIPELINE

includes investigational RNAi therapeutics for rare genetic, cardio-metabolic, hepatic infectious diseases, and CNS diseases.

We’re proud to be named a

TOP PLACE TO WORK

for the 4th year in a row!

 

 

NOW APPROVED IN THE U.S.

To those who say "impossible, impractical, unrealistic,"  we say:

CHALLENGE ACCEPTED

We're relentless in our pursuit of new treatments because patients shouldn't have to wait for hope.

Rose, living with a type of acute hepatic porphyria

We are developing a new class of innovative medicines called

RNAi THERAPEUTICS

that we believe have the potential to transform the lives of people living with rare diseases.

Dane, living with hATTR amyloidosis

We are focused on diseases for which treatment options are limited or simply don't exist.

OUR ROBUST PIPELINE

includes investigational RNAi therapeutics for rare genetic, cardio-metabolic, hepatic infectious diseases, and CNS diseases.

LATEST NEWS

>1k
Employees
7
Programs Currently in Clinical Development
1
Approved Product*
>20k
Doses Administered
>25
Clinical Studies to Date
>4yrs
Longest Duration of Exposure

Updated as of November 2018
* In the U.S. and European Union

 
 
 
 
>1k
 
 
Employees
 
 
 
 
7
 
 
Programs Currently in
Clinical Development
 
 
 
 
1
 
 
Approved Product*
 
 
 
 
>20k
 
 
Doses Administered
 
 
 
 
>25
 
 
Clinical Studies
to Date
 
 
 
 
>4yrs
 
 
Longest Duration
of Exposure

Updated as of November 2018
* In the U.S. and European Union

OUR FOCUS ON PATIENTS

A Family Living with
 hATTR Amyloidosis

Rose & Colin: Living with
Acute Hepatic Porphyrias

Venkat: Living with Hemophilia

OUR

PIPELINE

Late Stage

Learn more about our investigational therapies in hereditary ATTR amyloidosis, hemophilia, and acute hepatic porphyrias.

Early Stage

 

Learn more about our investigational therapies across multiple disease areas.

WORK WITH US

Join Us

We are looking for innovators to join our team—help us realize our commitment to developing a new class of medicines for patients with limited or inadequate options.

View Job Openings >

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